This observational, post-authorization, long-term follow-up study aims to investigate the short and long-term effectiveness and safety of HEMGENIX in patients with hemophilia B. The study will also include a cohort of patients with hemophilia B treated with FIX prophylaxis to enable interpretation of relevant efficacy and safety findings of HEMGENIX.
Study Type
OBSERVATIONAL
Enrollment
500
HEMGENIX is a gene therapy medicinal product that aims to deliver a factor IX (FIX) gene expression cassette to the liver of patients with hemophilia B.
FIX prophylaxis therapy
American Thrombosis and Hemostasis Network
Rochester, New York, United States
RECRUITINGMedical University Vienna
Vienna, Austria
RECRUITINGBleeding Rate (all bleeds) - HEMGENIX Cohort
Annualised Bleeding Rate (ABR) for all bleeds
Time frame: During the 52 weeks following stable FIX expression (6 to 18 months) in the follow-up period and at Months 6 to: 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, and 180 after HEMGENIX treatment.
Bleeding Rate (specified bleed types) - HEMGENIX Cohort
ABR for spontaneous, FIX-treated, traumatic, and joint bleeds
Time frame: During follow-up period (52 weeks following stable FIX expression [6 to 18 months]), and at Months 6 to: 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, and 180 after HEMGENIX treatment.
Zero bleeds - HEMGENIX Cohort
Number of patients with zero bleeds
Time frame: During follow-up after HEMGENIX treatment, up to 180 months
Correlation analysis - HEMGENIX Cohort
Correlation analysis of ABR as a function of mean FIX activity.
Time frame: Over the 6 to: 18, 24, 36, 48, 60, 72, 96, 108, 120, 132, 144, 156, 168, and 180 months after HEMGENIX treatment.
Bleeding Rate - FIX Prophylaxis Cohort
ABR (total, spontaneous, FIX-treated, traumatic, and joint bleeds)
Time frame: In the period Months 0 to: 12, 18, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, and 180 Months.
FIX activity - HEMGENIX Cohort
Mean uncontaminated endogenous FIX activity (%)
Time frame: Day 1, Week 6, and Months 6, 12, 18, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, and 180 after HEMGENIX treatment.
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Aarhus Universitetshospital
Århus N, Denmark
RECRUITINGCentre Hospitalier Universitaire de Brest / CHU Morvan
Brest, France
RECRUITINGCentre Régional de Traitement de l'Hémophilie
Nantes, France
RECRUITINGCHU Nancy - Hôpital Brabois
Vandœuvre-lès-Nancy, France
RECRUITINGKlinik für Angiologie/ Hämostaseologie
Berlin, Germany
RECRUITINGUniversity of Clinic Bonn
Bonn, Germany
RECRUITINGKlinikum der Johann-Wolfgang Goethe Universitaet
Frankfurt, Germany
RECRUITINGHannover Medical School
Hanover, Germany
RECRUITING...and 2 more locations
Annualized consumption of FIX replacement therapy - HEMGENIX Cohort
Mean annualized consumption (IU/kg/Yr) of FIX replacement therapy (including FIX replacement for various clinical scenarios eg, prophylaxis, spontaneous bleeding events, medical procedures, surgery, and trauma).
Time frame: During the follow-up periods (52 weeks following stable FIX expression [6 to 18 months], & at Months 0 to: 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, and 180), & at yearly intervals (ie, at 0 to 1 yr, 1 to 2 yrs, etc, up to 14 to 15 yrs).
Annualized consumption of FIX replacement therapy - FIX Prophylaxis Cohort
Mean annualized consumption (IU/kg/Yr) of FIX replacement therapy (including FIX replacement for various clinical scenarios eg, prophylaxis, spontaneous bleeding events, medical procedures, surgery, and trauma).
Time frame: At Months 0 to: 12, 18, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, and 180.
Number of patients remaining free of previous continuous routine prophylaxis - HEMGENIX Cohort
Number and proportion of patients remaining free of previous continuous routine prophylaxis (FIX replacement therapy)
Time frame: At Months 6 to: 18, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, and 180 after HEMGENIX treatment.
Target joints occurrence - HEMGENIX Cohort
The annualized occurrence of target joints (defined as 3 or more spontaneous bleeding events into a single joint within a consecutive 6-month period).
Time frame: After HEMGENIX treatment, during the follow-up periods (52 weeks following stable FIX expression [6 to 18 months], and at Months 6 to: 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, and 180).
Target joints resolution - HEMGENIX Cohort
The percentage of resolution of target joints (target resolution is defined as a recorded target joint with 2 or fewer spontaneous bleeding events within a consecutive 12-month period).
Time frame: During the follow-up period, up to 180 months
Target joints - FIX Prophylaxis Cohort
The annualized occurrence of target joints
Time frame: From Months 0 to 12, 18, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, and 180.
Related Serious Adverse Events (SAEs) - number
Incidence (number) of related SAEs
Time frame: During 15 years of follow-up
Related SAEs - percent
Incidence (percent) of related SAEs
Time frame: During 15 years of follow-up
Adverse Events of Special Interest (AESIs) - number
Incidence (number) of AESIs
Time frame: During 15 years of follow-up
AESIs - percent
Incidence (percent) of AESIs
Time frame: During 15 years of follow-up